Intraperitoneal delivery of a novel drug‐like compound improves disease severity in severe and intermediate mouse models of Spinal Muscular Atrophy

por Osman, E. Y., Rietz, A., Kline, R. A., Cherry, J. J., Hodgetts, K. J., Lorson, C. L., Androphy, E. J.
Publicado 2019

CRISPR/Cas9 mediated generation of an ovine model for infantile neuronal ceroid lipofuscinosis (CLN1 disease)

por Eaton, S. L., Proudfoot, C., Lillico, S. G., Skehel, P., Kline, R. A., Hamer, K., Rzechorzek, N. M., Clutton, E., Gregson, R., King, T., O’Neill, C. A., Cooper, J. D., Thompson, G., Whitelaw, C. B., Wishart, T. M.
Publicado 2019